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Cure CMT4J is generating awareness and support for the development of gene therapy to address CMT4J. By moving this research forward, other valuable discoveries and potential applications in viral vector therapies could be made possibly impacting other diseases and improving the quality of life for countless others.

Our latest updates

October 20 at 7:00 p.m.: Join us for our first annual Dare to be Rare Gala, featuring delicious food and cocktails, live music, unique performances, and a silent auction for prizes such as vacation homes, sporting events and evenings out. Be there, and be rare!

8-pack ticket holders will receive a zebra
LED bracelet to show your stripes at the
event and for entry into an exclusive raffle
reserved just for bracelet holders.

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Little known fact: A group of zebras is called a "dazzle" and in medicine, the term “zebra” refers to a rare disease or condition.

CMT4J and the hope for treatment

CMT4J, or Charcot Marie Tooth Disease Type 4J, is an ultra-rare, severe, progressive neuropathy caused by a mutation on the FIG4 gene. CMT4J can cause profound, accelerating limb weakness and muscle atrophy, making it very difficult for patients to walk or even lift up their arms. Some patients experience difficulty breathing due to phrenic nerve injury in the diaphragm. CMT4J is often compared to a slower form of ALS (Lou Gehrig’s Disease).
At present there is no treatment or cure.

However, the gene involved in the CMT4J mutation is considered to be an ideal gene for use in gene therapy due to its size and makeup. Because CMT4J is considered to be a peripheral nerve disorder, it is expected that many of the nerve cells already involved would have the chance to regenerate and re-myelinate with gene therapy. In theory, gene therapy would not only halt disease progression, but would also reverse effects on damaged nerve cells.

About Talia

At just eleven years old, Talia Duff is known to many in her community. She is the bright light among fifth graders who love to encircle her at the lunch table or play her imaginative games at recess. She is a big reader, a budding musician, actor, tandem cyclist and sit-skier. Talia is the center of a very special family and community who help her to embrace all that life has to offer. She is also brave beyond her years, having endured countless medical procedures and interventions for most of her life. Learn more about Talia »

About Ethan

Ethan came into the world in July 2010, an adorable 9lb 11oz giant who spent his first hours quietly observing the world around him. He ate well, rolled over on time, and charmed his way into the hearts of everyone that met him. Even as a very young child Ethan had a special air around him, somehow both mischievous and innocent—and oh so very sweet. He was born with soft clubfeet and at his checkups providers appeared to have trouble eliciting reflexes in his legs, but not much was thought of it at the time and there were no developmental concerns. Learn more about Ethan »

About Jakeb

From pregnancy until about 5 years old, everything seemed normal with Jakeb. He hit all the developmental milestones when he should have. He joined all the little sports groups for soccer, football, basketball, and baseball, but he wasn't too interested in them. But he loved to dance and has a huge contagious personality ... Learn more about Jakeb »

Research Team

We are excited to announce that Dr. Jun Li is on board as a member of our team! Dr. Li is a tenured Professor of Neurology at Vanderbilt University School of Medicine. He is sub-specialized in Neuromuscular Diseases with a special interest in Charcot-Marie-Tooth (CMT) diseases and myelin biology, including pathogenesis and therapeutic development of CMT4J. He directs the Vanderbilt CMT Clinic, which has been recognized by the CMT Association as a CMT Center of Excellence.

Contact

Are you affected by CMT4J?
If you or someone you love has CMT4J, we'd love to hear from you! Our work relies on making connections with others affected by CMT4J. Please call or email us — we're eager to connect and include you in our efforts.

Cure CMT4J / The Talia Duff Foundation Inc. (Tax ID: 81-3019217) is a Massachusetts corporation. Federal tax exempt status as a public charity under Section 501(c)(3) has been approved by the U.S. Internal Revenue Service.